These bills mirror the proposed statutory language set out in the Goldwater Institute Policy Report, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment,” published on Feb. 11, 2014.
The states’ bills allow (but do not require) the manufacturer of an investigational drug, biologic or device to make their product available (with or without compensation from the patient) to eligible terminally ill patients once the product successfully completes Phase I of clinical development, and as long as it remains under investigation in a clinical trial. The bills do not require insurance companies to cover the cost of the investigational product. In addition, these bills protect physicians from losing their medical license if they recommend an investigational product to an eligible patient and contain provisions that make attempts to block access to these products a criminal offense.
Currently, FDA oversees compassionate use, also known as expanded access, and reviews requests for approving or denying patient access to investigational treatments on a case-by-case basis.
On top of these states’ bills, Rep. Morgan Griffith (R-Va.) recently introduced H.R. 4475, the “Compassionate Freedom of Choice Act.” The bill would prevent FDA from restricting the importation, manufacture and sale of investigational drugs, biologics and devices to treat terminally ill patients. This bill also extends liability protections to manufacturers (and others in the supply chain) and prevents FDA from requiring collection of clinical outcomes from patients using these investigational products.
Even if these state laws are enacted, as a practical matter, manufacturers will likely be reluctant to supply these products to physicians due to the uncertainty of how state law would interact with Federal law. Along with media attention on specific cases, these state bills will bring to the forefront discussions about new approaches to facilitating expanded access to patients.
Each state bill is moving through their respective legislatures. Arizona’s bill passed the Senate and the House, and unless vetoed by the governor, will be voted on for approval in November.